EA201991455A1 - Композиции и способы лечения дефицита альфа-1-антитрипсина - Google Patents

Композиции и способы лечения дефицита альфа-1-антитрипсина

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Publication number
EA201991455A1
EA201991455A1 EA201991455A EA201991455A EA201991455A1 EA 201991455 A1 EA201991455 A1 EA 201991455A1 EA 201991455 A EA201991455 A EA 201991455A EA 201991455 A EA201991455 A EA 201991455A EA 201991455 A1 EA201991455 A1 EA 201991455A1
Authority
EA
Eurasian Patent Office
Prior art keywords
compositions
methods
deficiency
antitripsin
treating alpha
Prior art date
Application number
EA201991455A
Other languages
English (en)
Russian (ru)
Inventor
Сёбу Одате
Уолтер Стрэппс
Рейнальд Майкл Лескарбо
Original Assignee
Интеллиа Терапьютикс, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Интеллиа Терапьютикс, Инк. filed Critical Интеллиа Терапьютикс, Инк.
Publication of EA201991455A1 publication Critical patent/EA201991455A1/ru

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • A—HUMAN NECESSITIES
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • C12N9/14—Hydrolases (3)
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    • C12N2310/00—Structure or type of the nucleic acid
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EA201991455A 2016-12-22 2017-12-21 Композиции и способы лечения дефицита альфа-1-антитрипсина EA201991455A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662438219P 2016-12-22 2016-12-22
PCT/US2017/067800 WO2018119182A1 (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

Publications (1)

Publication Number Publication Date
EA201991455A1 true EA201991455A1 (ru) 2020-01-15

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EA201991455A EA201991455A1 (ru) 2016-12-22 2017-12-21 Композиции и способы лечения дефицита альфа-1-антитрипсина

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US (2) US11549107B2 (es)
EP (1) EP3559232A1 (es)
JP (2) JP7383478B2 (es)
KR (1) KR102551664B1 (es)
CN (2) CN110382697B (es)
AU (2) AU2017379073B2 (es)
BR (1) BR112019012825A2 (es)
CA (1) CA3047415A1 (es)
CO (1) CO2019007776A2 (es)
EA (1) EA201991455A1 (es)
IL (1) IL267448A (es)
MX (1) MX2019007594A (es)
NZ (1) NZ754739A (es)
PH (1) PH12019501457A1 (es)
SG (1) SG10202106412RA (es)
TW (1) TW201833331A (es)
WO (1) WO2018119182A1 (es)
ZA (1) ZA202007632B (es)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
CA3077413A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. Formulations
WO2020006708A1 (en) * 2018-07-04 2020-01-09 Yale University Compositions and methods for enhancement of homology-directed repair mediated precise gene editing by programming dna repair with a single rna-guided endonuclease
WO2020082047A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey
JP7472121B2 (ja) * 2018-10-18 2024-04-22 インテリア セラピューティクス，インコーポレーテッド アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法
US20220160847A1 (en) * 2020-11-20 2022-05-26 Mark Egly Method of preventing and/or treating a plurality of diseases
EP4259792A1 (en) 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination

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KR20160102056A (ko) * 2013-12-26 2016-08-26 더 제너럴 하스피탈 코포레이션 멀티플렉스 가이드 ｒｎａ
JP7068821B2 (ja) * 2014-12-03 2022-05-17 アジレント・テクノロジーズ・インク 化学修飾を有するガイドｒｎａ
CN107787367B (zh) * 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna
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MX2018005332A (es) * 2015-11-06 2018-11-09 Crispr Therapeutics Ag Materiales y metodos para tratamiento de la enfermedad de almacenamiento de glucogeno tipo 1a.
JP6932698B2 (ja) * 2015-12-01 2021-09-08 クリスパー・セラピューティクス・アクチェンゲゼルシャフトＣＲＩＳＰＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＡＧ アルファ１アンチトリプシン欠乏症の治療のための材料および方法
EP3433364A1 (en) * 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2020082047A1 (en) * 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey

Also Published As

Publication number Publication date
NZ754739A (en) 2024-01-26
ZA202007632B (en) 2023-03-29
IL267448A (en) 2019-08-29
SG10202106412RA (en) 2021-07-29
JP2020501582A (ja) 2020-01-23
MX2019007594A (es) 2019-12-16
PH12019501457A1 (en) 2020-03-09
US11549107B2 (en) 2023-01-10
JP7383478B2 (ja) 2023-11-20
WO2018119182A1 (en) 2018-06-28
TW201833331A (zh) 2018-09-16
KR102551664B1 (ko) 2023-07-05
EP3559232A1 (en) 2019-10-30
CN117737062A (zh) 2024-03-22
CA3047415A1 (en) 2018-06-28
AU2024201464A1 (en) 2024-03-28
US20230212575A1 (en) 2023-07-06
KR20190100943A (ko) 2019-08-29
BR112019012825A2 (pt) 2019-11-26
AU2017379073B2 (en) 2023-12-14
US20190316129A1 (en) 2019-10-17
CO2019007776A2 (es) 2019-10-09
JP2023075164A (ja) 2023-05-30
AU2017379073A1 (en) 2019-07-11
CN110382697A (zh) 2019-10-25
CN110382697B (zh) 2023-12-29

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